Journal
EXPERT OPINION ON BIOLOGICAL THERAPY
Volume 17, Issue 7, Pages 837-851Publisher
TAYLOR & FRANCIS LTD
DOI: 10.1080/14712598.2017.1323862
Keywords
Human umbilical cord blood; multipotency; ALS; treatment; neuroprotection
Funding
- Center of Excellence for Aging and Brain Repair at the University of South Florida
- Department of Neurosurgery and Brain Repair at the University of South Florida
- National Institutes of Health [1R01 NS090962-01]
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Introduction: Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease characterized by progressive motor neuron degeneration in the brain and spinal cord. Treatment options are limited due to the complexity of underlying disease factors. Cell therapy, using human umbilical cord blood (hUCB) cells may be a promising new treatment for ALS, mainly by providing a protective microenvironment for motor neuron survival.Areas covered: Composition, in vitro and in vivo differentiation of hUCB cells, and the advantages of cord blood as a source of transplant cells are discussed. A brief history of hUCB in treatment of an ALS animal model and the feasibility of these cells in therapy for ALS patients is provided. Current ALS clinical trials are also deliberated.Expert opinion: Among multiple advantages, hUCB cells' production of various anti-inflammatory/growth/trophic factors makes them an attractive cell source for ALS therapy. Biodistribution and optimal hUCB cell dose for transplantation have been determined in preclinical studies. Repeated intravenous cell doses during disease progression may be the best approach for cell-based ALS treatment. Accumulated evidence shows the efficacy of naive or genetically modified MNC hUCB cells in the treatment of ALS and provide a superior basis for the development of clinical trials in the near future.
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